BioPharm International - September 2022

BioPharm International - September 2022

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18 BioPharm International ® Emerging Therapies 2022 eBook T he field of cell and gene therapy has deliv- ered personalized and, in some cases, cura- tive therapeutics for various conditions in- cluding cancer and monogenetic disorders. Cell and gene therapies have been studied in various forms for decades, primarily in academia, and in re- cent years have gained traction in treating real-world conditions. The tide began to change in cell therapy about a decade ago with the treatment of the first pa- tients with CTL-019, the first chimeric antigen recep- tor T cells (CAR-T) approved by FDA, now known as Kymriah, at the University of Pennsylvania to treat acute lymphoblastic leukemia (ALL). And in gene therapy, Zolgensma took center stage for its ability to provide children with certain forms of spinal mus- cular atrophy (SMA), many times lethal by age four, a potentially curative treatment. As the pace of commercial approvals begins to gain steam, there are key questions about how to ef fi- ciently develop and, potentially more importantly, manufacture these therapeutics at a cost that bal- ances revenue with affordability. It's well known that cell and gene therapies are driving massive innova- tion in medicine. According to a report, the market for cell and gene therapies is expected to reach more than $36 billion by 2027, driven by the increasing number of clinical and commercial cellular and gene therapy products (1). While cell and gene therapy approvals are increas- ing and moving into larger indications (e.g., the de- velopment of a cell therapy for multiple myeloma) challenges remain. Many therapeutic developers both large and small lack critical expertise to effi- ciently advance cell and gene therapies to clinic and commercial production. These gaps span the devel- opment journey, from how to structure preclinical st udies t h rough chem ist r y, ma nufact ur ing, a nd controls (CMC) filings and regulatory submissions. Compounding a complex cell and gene therapy devel- opment framework is its fragmented nature, which The Road to Personalized Medicine: Reimagining Drug Manufacturing Matt Hewitt is executive director, Scientific Services Cell and Gene Therapy at Charles River. For cell and gene therapies to reach their full potential, changes in manufacturing must be explored. ANDRII YAL ANSKYI - STOCK.ADOBE.COM

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